自闭症
类有机物
表观基因组
神经科学
自闭症谱系障碍
生物
转录组
人脑
大脑发育
药物开发
生物信息学
计算生物学
医学
基因
遗传学
DNA甲基化
基因表达
精神科
药品
药理学
作者
Alexa Rabeling,Mubeen Goolam
出处
期刊:Gene Therapy
[Springer Nature]
日期:2022-07-05
卷期号:30 (9): 659-669
被引量:10
标识
DOI:10.1038/s41434-022-00356-z
摘要
Autism spectrum disorders (ASDs) are a set of disorders characterised by social and communication deficits caused by numerous genetic lesions affecting brain development. Progress in ASD research has been hampered by the lack of appropriate models, as both 2D cell culture as well as animal models cannot fully recapitulate the developing human brain or the pathogenesis of ASD. Recently, cerebral organoids have been developed to provide a more accurate, 3D in vitro model of human brain development. Cerebral organoids have been shown to recapitulate the foetal brain gene expression profile, transcriptome, epigenome, as well as disease dynamics of both idiopathic and syndromic ASDs. They are thus an excellent tool to investigate development of foetal stage ASDs, as well as interventions that can reverse or rescue the altered phenotypes observed. In this review, we discuss the development of cerebral organoids, their recent applications in the study of both syndromic and idiopathic ASDs, their use as an ASD drug development platform, as well as limitations of their use in ASD research.
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